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News: European Medicines Agency publishes first review of orphan designation

COMP, Last updated: 17/09/2010
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European Medicines Agency workshop on methodological aspects of clinical trials for efficacy evaluation in small populations

London, UK, 22/10/2002, Last updated: 12/11/2002
List item

EU/3/20/2391: Public summary of opinion on orphan designation: Idursulfase for the treatment of mucopolysaccharidosis type II (Hunter's syndrome) (PDF/172.91 KB) (updated)

Adopted
First published: 18/05/2021
Last updated: 27/01/2023
EMADOC-628903358-4024
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EU/3/12/1052: Public summary of opinion on orphan designation: Humanised monoclonal IgG4 antibody against tissue-factor-pathway inhibitor for the treatment of haemophilia A (PDF/229.63 KB)

First published: 16/11/2012
Last updated: 14/12/2022
EMA/COMP/619502/2012
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EU/3/17/1940: Public summary of opinion on orphan designation: Concizumab for the treatment of haemophilia B (PDF/179.66 KB)

Adopted
First published: 12/01/2018
Last updated: 14/12/2022
EMA/698762/2017
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EU/3/20/2370: Public summary of opinion on orphan designation: Tremelimumab for the treatment of hepatocellular carcinoma (PDF/183.38 KB)

First published: 21/05/2021
Last updated: 09/12/2022
EMADOC-628903358-3728
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EU/3/12/1091: Public summary of opinion on orphan designation: Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene for treatment of beta thalassaemia int... (PDF/226.3 KB)

Adopted
First published: 01/03/2013
Last updated: 30/11/2022
EMA/COMP/2193/2013 Rev.2
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Agenda of the COMP meeting 8-10 November 2022 (PDF/229.13 KB)

Draft
First published: 07/11/2022
Last updated: 08/11/2022
EMA/COMP/808207/2022 Corr.1
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EU/3/20/2322: Public summary of opinion on orphan designation: 6-((3S,4S)-4-Methyl-1-(pyrimidin-2-yl-methyl)pyrrolidin-3-yl)-3-tetrahydropyran-4-yl-7H-imidazo(1,5-a)pyrazin-8-one for the treatment of sickle cell disease (PDF/234.31 KB)

Adopted
First published: 25/11/2020
Last updated: 08/11/2022
EMADOC-628903358-2749
List item

EU/3/20/2329: Public summary of opinion on orphan designation: Infigratinib for the treatment of cholangiocarcinoma (PDF/199.99 KB)

Adopted
First published: 25/11/2020
Last updated: 08/11/2022
EMADOC-628903358-2757
List item

EU/3/05/315: Public summary of positive opinion for orphan designationof (2S)-2-[(4R)-2-oxo-4-propyltetrahydro-1H-pyrrol-1-yl] butanamidefor the treatment of progressive myoclonic epilepsies (PDF/298.5 KB)

Adopted
First published: 18/08/2008
Last updated: 08/11/2022
EMEA/COMP/247530/2005 Rev.1
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Minutes of the COMP meeting on 12-14 July 2022 (PDF/552.15 KB)

First published: 12/09/2022
Last updated: 21/10/2022
EMA/COMP/646322/2022 Corr.1
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EU/3/15/1599: Public summary of opinion on orphan designation: Adeno-associated viral vector serotype rh10 containing the human factor IX gene for the treatment of haemophilia B (PDF/204.79 KB)

Adopted
First published: 03/02/2016
Last updated: 11/10/2022
EMA/COMP/776835/2015
List item

Template - Translations required with the submission of an application for orphan medicinal product designation (DOC/71 KB)

First published: 10/12/2009
Last updated: 06/10/2022
List item

EU/3/12/1002: Public summary of opinion on orphan designation: Adenovirus-associated vector containing human Fas-c gene for the treatment of glioma (PDF/213.68 KB)

Adopted
First published: 04/07/2012
Last updated: 26/09/2022
EMA/COMP/268035/2012
List item

EU/3/17/1926: Public summary of opinion on orphan designation : Ofranergene obadenovec for the treatment of ovarian cancer (PDF/194.9 KB)

First published: 08/01/2018
Last updated: 26/09/2022
EMA/632137/2017
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EU/3/14/1382: Public summary of positive opinion for orphan designation: Allogeneic CD34+ cells expanded ex vivo with an aryl hydrocarbon receptor antagonist for the treatment of acute lymphoblastic leukaemia (PDF/223.74 KB)

Adopted
First published: 26/02/2015
Last updated: 16/09/2022
EMA/COMP/724271/2014
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EU/3/10/752: Public summary of positive opinion for Velaglucerase alfa for the treatment of Gaucher disease (PDF/181.91 KB)

Adopted
First published: 23/06/2010
Last updated: 09/09/2022
EMA/COMP/169787/2010 Rev. 1
List item

Recommendation for maintenance of orphan designation at the time of marketing authorisation for Vpriv (velaglucerase alfa) for the treatment of Gaucher disease (PDF/116.32 KB)

Adopted
First published: 13/09/2010
Last updated: 09/09/2022
EMA/COMP/433831/2010
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Recommendation for maintenance of orphan designation at the time of marketing authorisation: Revestive (teduglutide) for the treatment of short-bowel syndrome (PDF/111.78 KB)

Adopted
First published: 26/09/2012
Last updated: 09/09/2022
EMA/COMP/452266/2012
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EU/3/01/077: Public summary of positive opinion for orphan designation of [gly2]-recombinant human glucagon-like peptide for the treatment of short-bowel syndrome (PDF/193.8 KB)

Adopted
First published: 15/05/2009
Last updated: 09/09/2022
EMA/COMP/247424/2008 Rev.1
List item

EU/3/11/898: Public summary of opinion on orphan designation: Sirolimus for the treatment of chronic non-infectious uveitis (PDF/213.47 KB)

Adopted
First published: 21/09/2011
Last updated: 30/08/2022
EMA/COMP/508827/2011
List item

EU/3/13/1205: Public summary of opinion on orphan designation: Human monoclonal antibody against human interleukin 13 for the treatment of eosinophilic oesophagitis (PDF/195.1 KB)

Adopted
First published: 02/12/2013
Last updated: 30/08/2022
EMA/COMP/637681/2013 Rev. 1
List item

EU/3/18/2009: Public summary of opinion on orphan designation: Autologous dendritic cells pulsed with killed ovarian cancer cells and matured by TLR3 ligand ex vivo for the treatment of ovarian cancer (PDF/208.29 KB)

Adopted
First published: 17/05/2018
Last updated: 17/08/2022
EMA/198204/2018
List item

Kalydeco : Orphan maintenance assessment report (post-authorisation) (II-0089) (PDF/394.32 KB)

Adopted
First published: 12/05/2021
Last updated: 10/08/2022
EMA/OD/0000042076

2296 results

News: European Medicines Agency publishes first review of orphan designation

European Medicines Agency workshop on methodological aspects of clinical trials for efficacy evaluation in small populations

EU/3/20/2391: Public summary of opinion on orphan designation: Idursulfase for the treatment of mucopolysaccharidosis type II (Hunter's syndrome) (PDF/172.91 KB) (updated)

EU/3/12/1052: Public summary of opinion on orphan designation: Humanised monoclonal IgG4 antibody against tissue-factor-pathway inhibitor for the treatment of haemophilia A (PDF/229.63 KB)

EU/3/17/1940: Public summary of opinion on orphan designation: Concizumab for the treatment of haemophilia B (PDF/179.66 KB)

EU/3/20/2370: Public summary of opinion on orphan designation: Tremelimumab for the treatment of hepatocellular carcinoma (PDF/183.38 KB)

EU/3/12/1091: Public summary of opinion on orphan designation: Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene for treatment of beta thalassaemia int... (PDF/226.3 KB)

Agenda of the COMP meeting 8-10 November 2022 (PDF/229.13 KB)

EU/3/20/2322: Public summary of opinion on orphan designation: 6-((3S,4S)-4-Methyl-1-(pyrimidin-2-yl-methyl)pyrrolidin-3-yl)-3-tetrahydropyran-4-yl-7H-imidazo(1,5-a)pyrazin-8-one for the treatment of sickle cell disease (PDF/234.31 KB)

EU/3/20/2329: Public summary of opinion on orphan designation: Infigratinib for the treatment of cholangiocarcinoma (PDF/199.99 KB)

EU/3/05/315: Public summary of positive opinion for orphan designationof (2S)-2-[(4R)-2-oxo-4-propyltetrahydro-1H-pyrrol-1-yl] butanamidefor the treatment of progressive myoclonic epilepsies (PDF/298.5 KB)

Minutes of the COMP meeting on 12-14 July 2022 (PDF/552.15 KB)

EU/3/15/1599: Public summary of opinion on orphan designation: Adeno-associated viral vector serotype rh10 containing the human factor IX gene for the treatment of haemophilia B (PDF/204.79 KB)

Template - Translations required with the submission of an application for orphan medicinal product designation (DOC/71 KB)

EU/3/12/1002: Public summary of opinion on orphan designation: Adenovirus-associated vector containing human Fas-c gene for the treatment of glioma (PDF/213.68 KB)

EU/3/17/1926: Public summary of opinion on orphan designation : Ofranergene obadenovec for the treatment of ovarian cancer (PDF/194.9 KB)

EU/3/14/1382: Public summary of positive opinion for orphan designation: Allogeneic CD34+ cells expanded ex vivo with an aryl hydrocarbon receptor antagonist for the treatment of acute lymphoblastic leukaemia (PDF/223.74 KB)

EU/3/10/752: Public summary of positive opinion for Velaglucerase alfa for the treatment of Gaucher disease (PDF/181.91 KB)

Recommendation for maintenance of orphan designation at the time of marketing authorisation for Vpriv (velaglucerase alfa) for the treatment of Gaucher disease (PDF/116.32 KB)

Recommendation for maintenance of orphan designation at the time of marketing authorisation: Revestive (teduglutide) for the treatment of short-bowel syndrome (PDF/111.78 KB)

EU/3/01/077: Public summary of positive opinion for orphan designation of [gly2]-recombinant human glucagon-like peptide for the treatment of short-bowel syndrome (PDF/193.8 KB)

EU/3/11/898: Public summary of opinion on orphan designation: Sirolimus for the treatment of chronic non-infectious uveitis (PDF/213.47 KB)

EU/3/13/1205: Public summary of opinion on orphan designation: Human monoclonal antibody against human interleukin 13 for the treatment of eosinophilic oesophagitis (PDF/195.1 KB)

EU/3/18/2009: Public summary of opinion on orphan designation: Autologous dendritic cells pulsed with killed ovarian cancer cells and matured by TLR3 ligand ex vivo for the treatment of ovarian cancer (PDF/208.29 KB)

Kalydeco : Orphan maintenance assessment report (post-authorisation) (II-0089) (PDF/394.32 KB)